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Showing 1–24 of 406 matching trials from the live ClinicalTrials.gov search.
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Terminated No phase listed Observational

Longitudinal and Biological Study of Childhood Disintegrative Disorder

NCT00004458
View directory record Official record
Conditions
Pervasive Child Development Disorders, Autism
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 9 Years
Enrollment
220 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
2
States / cities
New Haven, Connecticut • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 7:41 PM EDT
Completed Phase 2 Interventional

Phase II Pilot Study of Cyclophosphamide and Rabbit Anti-Thymocyte Globulin as Salvage Therapy in Patients With Diffuse Systemic Sclerosis

NCT00016458
View directory record Official record
Conditions
Systemic Sclerosis
Interventions
anti-thymocyte globulin, cyclophosphamide
Drug
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 64 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
3
States / cities
Denver, Colorado • Durham, North Carolina • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 2, 2008 · Synced Jul 21, 2026, 7:41 PM EDT
Completed Phase 3 Interventional Results available

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

NCT02432144
View directory record Official record
Conditions
Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
Interventions
UX003
Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
5 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
4
States / cities
Oakland, California • Orange, California • Minneapolis, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2020 · Synced Jul 21, 2026, 7:41 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542
View directory record Official record
Conditions
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions
Family Centered pediatric palliative care for family caregivers of children with rare diseases.
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
12 Months to 99 Years
Enrollment
480 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced Jul 21, 2026, 7:41 PM EDT
Completed Not applicable Interventional

Randomized Study of Growth Hormone on Bone Mineral Density in Patients With Adult Onset Growth Hormone Deficiency

NCT00006394
View directory record Official record
Conditions
Osteoporosis, Growth Hormone Deficiency
Interventions
growth hormone
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1997
U.S. locations
7
States / cities
Boston, Massachusetts • Rochester, Minnesota • St Louis, Missouri + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 7:41 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

NCT03041324
View directory record Official record
Conditions
Mucopolysaccharidosis II, MPS II
Interventions
SB-913
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
5 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 24, 2022 · Synced Jul 21, 2026, 7:41 PM EDT
Active, not recruiting Phase 2 Interventional

A Study Evaluating the Efficacy and Safety of Biomarker-Driven Therapies in Patients With Persistent or Recurrent Rare Epithelial Ovarian Tumors

NCT04931342
View directory record Official record
Conditions
Ovarian Cancer
Interventions
Ipatasertib, Cobimetinib, Trastuzumab Emtansine, Atezolizumab, Bevacizumab, Paclitaxel, Giredestrant, Abemaciclib, Inavolisib, Palbociclib, Letrozole, Olaparib, Luteinizing Hormone-Releasing Hormone (LHRH) Agonists, Cyclophosphamide
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older · Female only
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
8
States / cities
San Francisco, California • St Louis, Missouri • Charlotte, North Carolina + 5 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jul 21, 2026, 7:41 PM EDT
Completed Phase 1 Interventional

Pilot Study of Budesonide for Patients With Primary Sclerosing Cholangitis

NCT00004842
View directory record Official record
Conditions
Cholangitis, Sclerosing, Liver Cirrhosis, Biliary
Interventions
budesonide
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
18 Years to 70 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 1999
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Jul 21, 2026, 7:41 PM EDT
Suspended Phase 1Phase 2 Interventional

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

NCT04771416
View directory record Official record
Conditions
Leukodystrophy, Globoid Cell
Interventions
PBKR03
Biological
Lead sponsor
Gemma Biotherapeutics
Industry
Eligibility
1 Month to 9 Months
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
4
States / cities
Chicago, Illinois • New York, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Jul 21, 2026, 7:41 PM EDT
Active, not recruiting Phase 2 Interventional

A Phase 2 Study of T-DXd in Patients With Selected HER2 Expressing Tumors

NCT04482309
View directory record Official record
Conditions
Part 1: Bladder, Biliary Tract, Cervical, Endometrial, Ovarian, Pancreatic Cancer, Rare Tumors, Any Tumor Type Excluding Breast, Gastric, Colorectal Cancer, Part 2: HER2 Expressing/Amplified Solid Tumors Excluding Breast, Gastric, Colorectal Cancer
Interventions
Trastuzumab deruxtecan
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 120 Years
Enrollment
477 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
12
States / cities
Duarte, California • Santa Rosa, California • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2026 · Synced Jul 21, 2026, 7:41 PM EDT
Completed Not applicable Interventional

Randomized Study of Photodynamic Therapy Using Dihematoporphyrin in Patients With Corneal Neovascularization

NCT00004430
View directory record Official record
Conditions
Corneal Neovascularization
Interventions
Dihematoporphyrin derivative, prednisolone, Laser surgery
Drug · Procedure
Lead sponsor
Medical College of Hampton Roads
Other
Eligibility
12 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 1998
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 24, 2015 · Synced Jul 21, 2026, 7:41 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Rare Diseases Clinical Research Network: Neurophysiological Correlates

NCT03077308
View directory record Official record
Conditions
Rett Syndrome, Preserved Speech Variant, Mecp2 Duplication Syndrome, Rett-related Disorders
Interventions
Auditory and Visual Event-related Potentials and EEG
Procedure
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
2 Years to 65 Years
Enrollment
185 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2021
U.S. locations
5
States / cities
Denver, Colorado • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2021 · Synced Jul 21, 2026, 7:41 PM EDT
Active, not recruiting No phase listed Observational

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871
View directory record Official record
Conditions
Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Interventions
SB-318, SB-913, SB-FIX
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
5
States / cities
Oakland, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated May 2, 2024 · Synced Jul 21, 2026, 7:41 PM EDT
Recruiting No phase listed Observational

Stakeholders of Rare Diseases Informing Values In Neuroethics

NCT07314736
View directory record Official record
Conditions
Rare Disorder, Disorder, Neurologic
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
385 participants
Timeline
2026 – 2031
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 21, 2026, 7:41 PM EDT
Available No phase listed Expanded access

Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease

NCT06931834
View directory record Official record
Conditions
Mitochondrial Disease, Chronic Kidney Disease
Interventions
Probucol
Drug
Lead sponsor
RiboNova Inc.
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 16, 2025 · Synced Jul 21, 2026, 7:41 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Study of Energy Expenditure in Infants With Ventricular Septal Defects

NCT00006272
View directory record Official record
Conditions
Heart Septal Defects, Ventricular
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
3 Months to 4 Months
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 1994
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 7:41 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Research for Individualized Therapeutics in Rare Genetic Disease

NCT05236595
View directory record Official record
Conditions
Rare Genetic Disease, Undiagnosed Diseases
Interventions
Individualized drug matching per genetic disease
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2026
U.S. locations
3
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Minneota, Minnesota
Source: ClinicalTrials.gov public record
Updated Jan 19, 2026 · Synced Jul 21, 2026, 7:41 PM EDT
Active, not recruiting Phase 2 Interventional Results available

BV-CHEP Chemotherapy for Adult T-cell Leukemia or Lymphoma

NCT03264131
View directory record Official record
Conditions
Lymphoma, Adult T-Cell Leukemia/Lymphoma, Lymphatic Diseases
Interventions
Brentuximab Vedotin, CHEP
Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
4
States / cities
Hollywood, Florida • Boston, Massachusetts • Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 9, 2025 · Synced Jul 21, 2026, 7:41 PM EDT
Not listed Phase 2 Interventional

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

NCT00004293
View directory record Official record
Conditions
Gaucher's Disease
Interventions
glucocerebrosidase
Drug
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 7:41 PM EDT
Recruiting No phase listed Observational

International Rare Histiocytic Disorders Registry (IRHDR)

NCT02285582
View directory record Official record
Conditions
Rare Histiocytic Disorders (RHDs), Erdheim-Chester Disease (ECD), Rosai-Dorfman Disease (RDD), Xanthogranuloma Family (XG), Indeterminate Dendritic Cell Histiocytosis, Malignant Histiocytic Neoplasm (MHN), ALK-positive Histiocytosis, Mixed Histiocytosis (MXH), Multicentric Reticulohistiocytoma (MRH), Necrobiotic Xanthogranuloma (NX)
Interventions
Registry study
Other
Lead sponsor
The Hospital for Sick Children
Other
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2028
U.S. locations
6
States / cities
Birmingham, Alabama • Los Angeles, California • Madera, California + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 23, 2025 · Synced Jul 21, 2026, 7:41 PM EDT
Completed No phase listed Observational

Pilot Study of Familial Nonsyndromal Mondini Dysplasia

NCT00004336
View directory record Official record
Conditions
Mondini Dysplasia
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
0 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jun 23, 2005 · Synced Jul 21, 2026, 7:41 PM EDT
Completed Phase 2 Interventional

Phase II Study of High-Dose Cyclophosphamide in Patients With Severe Autoimmune Hematologic Disease

NCT00010387
View directory record Official record
Conditions
Anemia, Hemolytic, Autoimmune, Felty Syndrome, Purpura, Thrombocytopenic, Autoimmune Diseases
Interventions
cyclophosphamide, filgrastim
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 9, 2008 · Synced Jul 21, 2026, 7:41 PM EDT
Completed Phase 2 Interventional Results available

Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies

NCT02358538
View directory record Official record
Conditions
Epilepsy
Interventions
Ganaxolone
Drug
Lead sponsor
Marinus Pharmaceuticals
Industry
Eligibility
2 Years to 18 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
9
States / cities
Phoenix, Arizona • Sacramento, California • San Francisco, California + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Jul 21, 2026, 7:41 PM EDT
Terminated Phase 2 Interventional

Durvalumab in Combination With Tremelimumab in Subjects With Advanced Rare Solid Tumors

NCT02938793
View directory record Official record
Conditions
Cancer, Rare Disease
Interventions
Durvalumab, Tremelimumab
Drug
Lead sponsor
Prisma Health-Upstate
Other
Eligibility
18 Years and older
Enrollment
92 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
1
States / cities
Greenville, South Carolina
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced Jul 21, 2026, 7:41 PM EDT